Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv) Patients Included in B3461028 and B3461045.

NCT05560555 | Completed Results

• 2 other identifiers: B3461104TRAMA
• Study Type: observational
• Target: 5
• Locations: 1 country
• Sites: 3

Brief Summary

A study of patients with hereditary transthyretin amyloidosis (ATTRv) and wild-type transthyretin amyloidosis (ATTRwt) that have been enrolled in B3461028 and B3461045 studies in Spain - exposed to tafamidis 61mg for ≥12 months with polyneuropathy (PN) have kept going to their multisystemic follow-ups (neuro/ophthalmo/gastrointestinal) ≥12 months.

Conditions

Hereditary Transthyretin Amyloidosis (ATTRv); Polyneuropathy

Outcome Measures

Primary Outcomes (1)

• Change in Neuropathy Impairment Score (NIS) at Month 12 for ATTRv

  NIS (Neuropathy Impairment Score) is a clinically important, sensitive measure of individual neurological function, assessing sensory function, reflexes, and muscle weakness. NIS score ranged from 0 to 244, with higher score indicating greater disability or impairment. The rate of change was calculated from last follow-up.

  Baseline and Month 12 (data collected and analyzed over 22 days)

Secondary Outcomes (33)

• Change in NIS for ATTRv

  Baseline, Month 6, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)

• Change in Neuropathy Impairment Score - Lower Limbs (NIS-LL) for ATTRv

  Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)

• Change in Norfolk Quality of Life- Diabetic Neuropathy (Norfolk QOL-DN) for ATTRv

  Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)

• Change in COMPASS-31 for ATTRv

  Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)

• Change in Familial Amyloid Polyneuropathy Specific Rasch-Built Overall Disability Scale (FAP-RODs) for ATTRv

  Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)

Study Arms (1)

Retrospective cohort ATTRv and ATTRwt patients enrolled in B3461028 and B3461045 studies in Spain

Drug: Tafamidis

Interventions

Tafamidis DRUG

61 milligrams (mg) as received in studies B3461028 and B3461045

Retrospective cohort ATTRv and ATTRwt patients enrolled in B3461028 and B3461045 studies in Spain

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Study population: retrospective cohort of patients with hereditary transthyretin amyloidosis (ATTRv) and wild-type transthyretin amyloidosis (ATTRwt) that have been enrolled in B3461028 and B3461045 studies in Spain - exposed to tafamidis 61mg for ≥12 months with polyneuropathy have kept going to their multisystemic follow-ups (neuro/ophthalmo/gastrointestinal) ≥12 months

You may qualify if:

• Treatment with tafamidis 61 mg ≥ 12 months
• Neurological follow up ≥ 12 months
• Diagnosis of transthyretin amyloidosis with polyneuropathy (ATTR-PN) based on one of the following:
• Amplitude reduction in, at least, 2 nerves under normal value, excluding median nerve OR 50% amplitude reduction in, at least, 2 nerves on the basal value of the patient, excluding median nerve OR 2 abnormal tests detecting thin fibers alterations (through Sudo scan, RR Interval analysis, etc..)

You may not qualify if:

• Treatment with tafamidis 61 mg \< 12 months
• Neurological follow up \< 12 months
• Other diagnosis for polyneuropathy

Sponsors & Collaborators

• Pfizer: lead

Study Sites (3)

Hospital Universitari de Bellvitge

Barcelona, 08907, Spain

Location

Hospital Clinico San Carlos

Madrid, 28040, Spain

Location

Hospital Son Llatzer

Palma de Mallorca, 07198, Spain

Location

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Amyloidosis, Hereditary, Transthyretin-Related; Polyneuropathies

Interventions

tafamidis

Condition Hierarchy (Ancestors)

Peripheral Nervous System Diseases; Neuromuscular Diseases; Nervous System Diseases

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 26, 2022
• First Posted: September 29, 2022
• Study Start: October 24, 2022
• Primary Completion: November 15, 2022
• Study Completion: November 15, 2022
• Last Updated: May 13, 2024
• Results First Posted: May 13, 2024

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will not share

Locations

ES (3)